A Phase 2 Study of Ulixertinib for People With Histiocytic Neoplasms


Full Title

Phase 2 Trial of Ulixertinib for Patients with Histiocytic Neoplasms


Researchers want to see how well ulixertinib works in people with histiocytic neoplasms. Histiocytic neoplasms are rare diseases in which too many white blood cells called histiocytes build up in tissues and organs. This can cause damage to tissue or tumor(s) to form. The tumor(s) may be benign (not cancer) or malignant (cancer). There are different types of histiocytic neoplasms, including:

  • Erdheim-Chester disease
  • Langerhans cell histiocytosis
  • Rosai-Dorfman disease
  • Histiocytic sarcoma
  • Juvenile xanthogranuloma

The people in this study have a histiocytic neoplasm (tumor) that keeps growing even with treatment or threatens their health. The tumor may also have a change in a protein pathway called MAPK. The MAPK pathway plays a role in the making of proteins and in cell growth and survival.

Ulixertinib blocks a protein called ERK, which is related to the MAPK pathway. Blocking this protein may help slow or stop the growth of tumor cells. Ulixertinib is taken orally (by mouth).

Who Can Join

To join this study, there are a few conditions. You must:

  • Have a histiocytic neoplasm that keeps growing even with treatment or threatens your health.
  • Have completed prior histiocytic neoplasm treatments at least 4 weeks before taking ulixertinib.
  • Be able to walk and do routine activities for more than half the time you are awake.
  • Be age 18 or older.


For more information or to see if you can join this study, please call Dr. Eli Diamond’s office at 212-610-0243.




Phase II (phase 2)

Disease Status

Relapsed or Refractory




ClinicalTrials.gov ID